A Look at BioMarin's Potential Blockbuster and the Science Behind It All

BioMarin Pharmaceutical just put out an update relating to its lead achondroplasia asset, Vosoritide, and the company is up a couple of percentage points on the data. This reaction isn’t really representative of the drug’s underlying potential, however. The data reinforces an efficacy readout of previous releases, and establishes a dose protocol for a pivotal trial in this indication, set to kick off before the end of 2016. With this trial in mind, and ahead of its initiation, here’s a look at what the drug involves and what the potential patient population looks like.

Back to news